Animal models are vital tools in the development of therapies for orphan diseases, given the small populations of patients available to evaluate the therapies. Here, Sepodes and colleagues from the European Medicines Agency's Committee for Orphan Medicinal Products harness their experience to provide an overview of the animal models used to support regulatory applications for metabolic, neuromuscular and ophthalmological rare diseases.
- Guillaume Vaquer
- Frida Rivière Dannerstedt
- Bruno Sepodes
